Data Suggest Denufosol May Hold Promise as an Early Intervention Treatment for Cystic Fibrosis

DURHAM, N.C.--(BUSINESS WIRE)--May 19, 2010 - Inspire
Pharmaceuticals, Inc. (NASDAQ: ISPH) announced today that Inspire
scientists and collaborators will present data on denufosol
tetrasodium, an investigational therapy for cystic fibrosis (CF),
during an oral presentation at the American Thoracic Society (ATS)
2010 International Conference May 14 - 19, 2010 in New Orleans, LA.
The data suggest that denufosol, an inhaled ion channel regulator,
has properties that allow it to reach and improve lung function in
the small airways, which may support its potential as an early
intervention therapy.

“It is well known that cystic fibrosis lung disease begins
in the small airways; however, there has been limited research into
treatments that reach these areas of the lungs,” said Felix
Ratjen, M.D., Ph.D., Professor of Pediatrics and Division Chief,
Respiratory Medicine, University of Toronto, and lead principal
investigator of TIGER-2. “Since pathological changes in small
airways of CF patients are seen as early as six months of age, and
mandatory newborn screenings in all 50 states means earlier
diagnoses, there is strong need for early intervention treatments
in the CF community right now.”

Inspire's oral presentation, “Denufosol Tetrasodium
- An Investigational Inhaled Therapy with Aerosol and
Pharmacokinetic Properties Suitable for Early Intervention in CF
Lung Disease” (T. Navratil, C. Evans, A. Schaberg, F.
Johnson, T. Durham, L. Smiley, N. Herje, B. Burke, C.L. Ren, F.
Ratjen, R.B. Moss, F.J. Accurso), will be presented in the Morial
Convention Center, Room 391-392 (Third Level) during the session,
“D94 Cystic Fibrosis: From Clinic to Lab and Back,” on
Wednesday, May 19 from 3:00 - 3:15 pm CST. The presentation
highlights clinical data from Inspire's first Phase 3 clinical
trial with denufosol, TIGER-1, and data from in vitro
testing. Such in vitro data suggests that denufosol, as a
result of its aerosol and chemical characteristics, has the
potential to reach the small airways of the lungs, where CF lung
disease begins. The potential of denufosol to reach the lung's
small airways was supported by a post-hoc analysis of the TIGER-1
trial, which showed a significant improvement relative to placebo
in FEF25%-75% (Forced Expiratory Flow) in a subgroup of
patients (n=329) with baseline FEV1 (Forced Expiratory
Volume in One Second) ‰¤110% predicted normal
(p=0.025). In the TIGER-1 trial, following inhalation, there was
little to no evidence of systemic exposure to denufosol and no
evidence of accumulation as measured by plasma levels. In the
trial, denufosol was well-tolerated and had a favorable safety
profile.

“The analysis from this study suggests that denufosol may
have the potential to target the lung's small airways, with little
to no systemic exposure, making it a potentially promising
treatment for early intervention in CF,” said M. Lynn Smiley,
M.D., Chief Medical Officer of Inspire. “In developing
denufosol, we hope to change the course of CF by providing an early
intervention strategy to lessen the destructive and irreversible
effects of the disease, regardless of CF genotypes.”

The Company also provided two additional poster presentations
given at the ATS Conference. The poster presentation,
“Concomitant Medication Use in Patients with Mild
Cystic Fibrosis Lung Disease Assigned to Placebo in a Phase 3
Clinical Trial of Denufosol (Study
08-108/TIGER-1)” (T. Durham, T. Navratil, A.
Schaberg, C. Deans, L. Smiley, N. Herje, G. Retsch-Bogart, F.J.
Accurso), examined the concomitant medication use in young,
relatively mild CF patients to treat respiratory conditions
resulting from CF, which involved treatment with inhaled,
intranasal, oral and IV medications. Data analyzed from a sample of
175 CF patients who received placebo in the TIGER-1 trial showed
that more than 72% of patients used at least five concomitant
medications for the treatment of their CF lung disease. New disease
modifying treatments that are targeted towards addressing the basic
defects of CF lung disease pathophysiology may reduce the need for
the extensive polypharmacy.

Inspire also presented a poster presentation entitled,
“Chronic Inhalation of Denufosol Tetrasodium Aerosol is
Non-Inflammatory in Rodent and Canine Respiratory Tract
Tissues” (M.S. Cowlen, R.S. Verhoeven), which showed
that in the required one-year and two-year pre-clinical studies,
denufosol did not increase background levels of pulmonary
inflammation compared with isotonic saline following daily
inhalation.

These presentations will be available on Inspire's website,
www.inspirepharm.com, following the conference.

About Denufosol Tetrasodium

Denufosol is a first-in-class ion channel regulator targeted as
an early intervention treatment that potentially corrects the ion
transport defect in patients with all CF genotypes. Denufosol is
designed to enhance airway hydration and mucociliary clearance by
increasing chloride secretion, inhibiting sodium absorption and
increasing ciliary beat frequency. These integrated pharmacological
actions and the potential to reach the small airways are key to
maintaining lung function and potentially delaying the progression
of lung disease.

Inspire is currently conducting its second Phase 3 clinical
trial with denufosol, TIGER-2.

About Inspire

Inspire is a biopharmaceutical company focused on researching,
developing and commercializing prescription pharmaceutical products
for ophthalmic and pulmonary diseases. Inspire's goal is to build
and commercialize a sustainable portfolio of innovative new
products based on its technical, scientific and commercial
expertise. The most advanced compounds in Inspire's clinical
pipeline are denufosol tetrasodium for cystic fibrosis and
PROLACRIA™ (diquafosol tetrasodium
ophthalmic solution) 2% for dry eye, which are both in Phase 3
development, and AZASITE® (azithromycin
ophthalmic solution) 1% for blepharitis, which is in Phase 2
development. Inspire receives revenues related to the promotion of
AZASITE for bacterial conjunctivitis, the co-promotion of
ELESTAT® (epinastine HCl ophthalmic solution)
0.05% for allergic conjunctivitis and royalties based on net sales
of RESTASIS® (cyclosporine ophthalmic
emulsion) 0.05% for dry eye. For more information, visit
www.inspirepharm.com.

Forward-Looking Statements

The forward-looking statements in this news release relating to
management's expectations and beliefs are based on preliminary
information and management assumptions. Specifically, no assurance
can be made with respect to: whether the properties or chemical
characteristics of denufosol may allow it to reach and improve lung
function in the small airways; whether the properties of denufosol
or improved lung function, if any, resulting from use of denufosol
may support its potential as an early intervention therapy; whether
the potential of denufosol to reach the lung's small airways will
be supported by any additional analyses or clinical studies;
whether additional analyses will suggest that use of denufosol
results in little to no systemic exposure; that use of denufosol
will lessen the destructive and irreversible effects of cystic
fibrosis lung disease, regardless of CF genotypes; that new disease
modifying treatments that are targeted toward addressing the basic
defects of CF lung disease pathophysiology may reduce the need for
extensive polypharmacy; and whether Inspire will successfully be
able to build and commercialize a sustainable portfolio of
innovative new products based on its technical, scientific and
commercial expertise. Such forward-looking statements are subject
to a wide range of risks and uncertainties that could cause results
to differ in material respects, including those relating to product
development, revenue, expense and earnings expectations, the
seasonality of ELESTAT, intellectual property rights, competitive
products, results and timing of clinical trials, success of
marketing efforts, the need for additional research and testing,
delays in manufacturing, funding, and the timing and content of
decisions made by regulatory authorities, including the U.S. Food
and Drug Administration. Further information regarding factors that
could affect Inspire's results is included in Inspire's filings
with the SEC. Inspire undertakes no obligation to publicly release
the results of any revisions to these forward-looking statements
that may be made to reflect events or circumstances after the date
hereof.

Drugs.com provides accurate and independent information on more than 24,000 prescription drugs, over-the-counter medicines and natural products. This material is provided for educational purposes only and is not intended for medical advice, diagnosis or treatment.